Ezutromid (SMT C1100) is a clinical stage compound that was developed for its ability to increase expression of utrophin (UTRN
), as a substitute for missing the dystrophin (DMD
) in Duchenne muscular dystrophy (DMD) patients [1
]. It is orally active. The aryl hydrocarbon receptor (AhR) has been reported as a molecular target for ezutromid, with ezutromid behaving as an AhR antagonist [2
]. Ezutromid and other AhR antagonists up-regulate utrophin, adding to the proposition that this pathway offers disease-modifying potential for DMD.